Clinical trials: Primary Hyperoxaluria Type 1
Primary Hyperoxaluria Type 1 (PH1)
Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)
The goal of the redePHine study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ABO-101 in participants with primary hyperoxaluria type 1 (PH1). The trial will consist of 2 Study Periods.
- Trial name
- NCT06839235
- Testing
- ABO-101
- Where
- 7 sites in United States, France and 3 more
- Volunteers
- 23 people
- Sponsor
- Arbor Biotechnologies
Primary Hyperoxaluria Type 1
A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
A phase 1 study of DCR-PH1 in patients with primary hyperoxaluria type 1 (PH1) to determine the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics (PD) effects of DCR-PH1 administered via-intravenous infusion (IV)
- Trial name
- NCT02795325
- Testing
- DCR-PH1
- Where
- 2 sites in Germany, Netherlands
- Volunteers
- 41 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Primary Hyperoxaluria Type 1 (PH1)
Treatment of Primary Hyperoxaluria Type 1 With Nedosiran
The purpose of this program is to provide participants with access to an investigational drug, nedosiran, for treatment of primary hyperoxaluria type 1 (PH1).
- Trial name
- NCT05993416
- Testing
- Nedosiran
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-B (NCT03905694)
- Testing
- Lumasiran
- Where
- 9 sites in United States, France and 3 more
- Volunteers
- 18 people
- Sponsor
- Alnylam Pharmaceuticals
Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis
National Registry of Rare Kidney Diseases
The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research.
- Conditions
- Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis, AHL Amyloidosis, AL Amyloidosis
- Trial name
- RaDaR (NCT06065852)
- Where
- United Kingdom
- Volunteers
- 35,000 people
- Sponsor
- UK Kidney Association
Primary Hyperoxaluria Type 3
Study to Evaluate Safety, Tolerability, PK and PD of DCR-PHXC in PH Type 3 Patients
The DCR-PHXC-104 study is designed to assess the safety, tolerability, and pharmacological parameters of a single dose of DCR-PHXC in Primary Hyperoxaluria Type 3 (PH3).
- Trial name
- PHYOX4 (NCT04555486)
- Testing
- DCR-PHXC, Sterile Normal Saline (0.9% NaCl)
- Where
- 6 sites in United States, Germany and 2 more
- Volunteers
- 6 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
Rare Kidney Stone Diseases
Monogenic Kidney Stone - Genetic Testing
This study will attempt to identify the specific gene (coded in the DNA) and changes (mutations) within that gene that are the cause of monogenic kidney stone disease.
- Trial name
- NCT03305835
- Where
- United States
- Volunteers
- 6,000 people
- Sponsor
- Mayo Clinic
Primary Hyperoxaluria Type 1 (PH1)
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-A (NCT03681184)
- Testing
- Lumasiran
- Where
- 17 sites in United States, France and 6 more
- Volunteers
- 39 people
- Sponsor
- Alnylam Pharmaceuticals
Primary Hyperoxaluria
Evaluation of the Efficacy of Stiripentol (Diacomit) as Monotherapy for the Treatment of Primary Hyperoxaluria
Evaluation of the efficacy of stiripentol (Diacomit) as monotherapy for the treatment of primary hyperoxaluria. Pilot clinical study, open, prospective and multicenter.
- Trial name
- NCT03819647
- Testing
- stiripentol (Diacomit)
- Where
- 3 sites in France
- Volunteers
- 15 people
- Sponsor
- Biocodex
Carcinoma, Renal Cell
Study of SU011248 in Patients With Advanced Kidney Cancer
To determine the objective tumor response of single-agent SU011248 at a dose of 50 mg orally once daily for 4 consecutive weeks and 2 weeks rest, repeated every 6 weeks in patients with metastatic Renal Cell Cancer (RCC).
- Trial name
- NCT00254540
- Testing
- SU011248 capsule
- Where
- 11 sites in Japan
- Volunteers
- 51 people
- Sponsor
- Pfizer
PH1, Primary Hyperoxaluria
An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the long-term safety and tolerability of lumasiran in participants with Primary Hyperoxaluria Type 1.
- Conditions
- PH1, Primary Hyperoxaluria, RNAi Therapeutic, siRNA
- Trial name
- NCT03350451
- Testing
- Lumasiran
- Where
- 9 sites in France, Germany and 3 more
- Volunteers
- 20 people
- Sponsor
- Alnylam Pharmaceuticals
Primary Hyperoxaluria
Study to Evaluate the Efficacy and Safety of Oxabact (OC5) in Patients With Primary Hyperoxaluria
The purpose of this study is to determine if Oxalobacter formigenes is effective at lowering urinary oxalate levels in patients with primary hyperoxaluria.
- Trial name
- NCT02012985
- Testing
- Oxabact OC5 capsules
- Where
- 8 sites in France, Germany and 1 more
- Volunteers
- 28 people
- Sponsor
- OxThera
Hyperoxaluria
Renal Protective Effect of ACEI and ARB in Primary Hyperoxaluria
This study will test the effectiveness of two medications: ACEI (angiotensin converting enzyme inhibitor)and ARB (angiotensin receptor blocker) in reducing the renal injury induced by hyperoxaluria in patients with Primary…
- Trial name
- NCT00280215
- Testing
- ACEI / Angiotensin converting enzyme inhibitor, ARB /Angiotensin Receptor Blocker
- Sponsor
- Mayo Clinic
Hyperoxaluria
Efficacy of Betaine for Reduction of Urine Oxalate in Patients With Type 1 Primary Hyperoxaluria
The aim of this study is to assess the efficacy and safety of betaine in reducing urine oxalate excretion of Type 1 Primary Hyperoxaluria (PHI) patients.
- Trial name
- NCT00283387
- Testing
- Betaine
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Mayo Clinic
Cystinosis, Primary Hyperoxaluria
Genetic Newborn Screening for Cystinosis and Primary Hyperoxaluria
In Germany parents of newborns are offered newborn screening (NBS) for 17 congenital diseases as a standard benefit of statutory health insurance. NBS in Germany is voluntary. Cystinosis and hyperoxaluria are very rare diseases.
- Trial name
- GENESIS (NCT05843851)
- Testing
- Diagnostic test
- Where
- Germany
- Volunteers
- 200,000 people
- Sponsor
- Cystinose Stiftung